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Genzyme Corp.

Latest From Genzyme Corp.

Analysis

In a recent interview, ProQR’s founder Daniel de Boer cited the enormous impact that fellow Dutch businessman Henri Termer played in getting his fledging biotech start-up off the ground – especially given the circumstances. Some 10 years ago, the then twenty-something budding biotech entrepreneur crossed the Atlantic on a chance that Termeer would answer what amounted to an unscheduled desk-knock at the rare disease pioneer’s office at Genzyme HQ in Cambridge, MA. He did – and the rest is history.

Analysis

How did a youthful vendor of IT services, faced with a personal exposure to crippling illness, attract some of the best minds in biotech to launch a company with a then-untested RNA technology that today is a contender for leadership in the rarified space of inherited retinal disease? The answer may lie in the ambitiously definitive name that 36-year-old founder Daniel de Boer gave to his enterprise with a mission: ProQR – an active synonym for finding cures.

News

Half of the newly selected imported drugs slated for fast track review in China are rare disease treatments, followed by antiviral drugs for influenza and HIV, reflecting key medical needs in the country.

News

bluebird bio is set to enter the EU market with the first gene therapy for transfusion-dependent β-thalassemia, after the European Medicines Agency reviewed its product in record time. Bluebird believes that the agency’s Priority Medicines (PRIME) and adaptive pathways programs have been instrumental in smoothing its path.